OBJECTIVES: Introduction of tyrosine kinase inhibitors (TKIs) has revolutionized the treatment of chronic myeloid leukemia (CML) but an unmet need still exists in patients receiving later lines of therapy. A review was conducted to understand the published evidence on the humanistic and economic burden associated with CML.

METHODS: Literature search of English publications from the last 20 years (2001-2020) was performed in Embase® and Medline® databases. Studies reporting health related quality of life (HRQoL), symptom burden and costs associated with CML patients were included.

RESULTS: Twenty-one studies reported humanistic burden, ten studies reported economic burden and two studies reported both humanistic together with economic burden. SF-36 and EORTC QLQ-C30 were the two most commonly used instruments assessing Quality of life (QoL). Physical function was one of the most severely impacted domain with females presenting with worse HRQoL profile than males. Fatigue, musculoskeletal pain, and disturbed sleep were the most frequently reported symptoms. Primary drivers of increased cost burden associated with treatment failure in later lines of therapies were medical services such as inpatient, emergency room, ambulatory and laboratory tests. In USA, average 1 year cost post TKI failure (adjusted to 2012 USD) for patients who failed first-line treatment was $78,677, which increased to $99,624 for patients who failed second-line treatment and was highest for patients failing third-line therapy ($181,029). Patients in later line of TKI therapies had significantly higher resource utilizations.

CONCLUSIONS: CML patients had significantly impaired HRQoL across all lines of therapies accompanied by increased symptom burden. Economic burden post-TKI failure increases with each sequential line of therapy. Results from the literature suggest the need for treatments that address long-term outcomes including reduced treatment resistance and improved Qol. This will further reduce the economic burden associated with the treatment of CML in later lines of therapy.